E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized Myasthenia Gravis |
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E.1.1.1 | Medical condition in easily understood language |
Myasthenia Gravis in patients aged 2 to 18 years who have generalized Muscle Weakness
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E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028417 |
E.1.2 | Term | Myasthenia gravis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the safety and tolerability of efgartigimod IV |
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E.2.2 | Secondary objectives of the trial |
To evaluate the immunogenicity of efgartigimod IV |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. The participant completed ARGX-113-2006, defined as: a. The participant reached EoT in trial ARGX-113-2006 and agreed to participate in the ARGX-113-2008 trial. b. The participant qualifies for retreatment in trial ARGX-113-2006, but cannot complete a Treatment Period (TP) and the required IP visits within the ARGX-113-2006 trial's timeframe (ie, between TPnV1 to IPnV4). 2. Either the participant or the participant’s legally acceptable representative (LAR) can understand the requirements of the trial and provide written informed consent/assent (including consent/assent for the use and disclosure of research-related health information), and demonstrates a willingness and ability to comply with the trial protocol procedures (including attending the required trial visits). 3. Contraceptive use by males and females must be consistent with local regulations for those participating in clinical trials and: a. Male participants agree not to donate sperm from the time the informed consent form (ICF) is signed until the end of the trial. b. Female adolescents of childbearing potential (FAOCBP; defined in Section 10.4.1.1 of the protocol ) must have a negative urine pregnancy test at trial entry (TP1V1 or IP0V1). The contraceptive requirements for male participants are described in Section 10.4.2.2. The contraceptive requirements for FAOCBP are described in Section 10.4.2.1. |
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E.4 | Principal exclusion criteria |
Participants are excluded from the trial if any of the following criteria apply: 1. FAOCBP: Pregnancy or lactation, or the participant intends to become pregnant during the trial or within 90 days after the last dose of investigational medicinal product (IMP) 2. Discontinued early from ARGX-113-2006 treatment due to: pregnancy, receiving prohibited medication, participating in another trial with an investigational product, or the occurrence of a life-threatening or an investigational medicinal product-related AE, as assessed by the investigator 3. A known hypersensitivity reaction to efgartigimod or any of its excipients 4. Received a live-attenuated vaccine fewer than 4 weeks before trial entry. 5. Any of the following medical conditions: a. Clinically significant uncontrolled chronic bacterial, viral, or fungal infection at trial entry b. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with accurate assessment of clinical symptoms of gMG or put the participant at undue risk |
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E.5 End points |
E.5.1 | Primary end point(s) |
- Incidence and severity of adverse events (AEs) - Incidence and severity of serious adverse events (SAEs) - Incidence and severity of adverse events of special interest (AESIs) - Changes in laboratory test results, vital signs, height and weight, and electrocardiogram (ECG) results |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Incidence and prevalence of antidrug antibodies (ADAs) against efgartigimod |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
No specific timepoints, during the entire study
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
- Tolerability - Immunogenicity |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 21 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Canada |
Georgia |
United States |
Austria |
Belgium |
France |
Germany |
Italy |
Netherlands |
Poland |
Spain |
United Kingdom |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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The end of the trial is defined as the date of the last visit of the last participant in the ARGX-113-2008 trial. A participant is considered to have completed the trial if he/she completed the end of trial (EoT) visit and safety follow-up (SFU) visit |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | 10 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 4 |